Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for…
Articles by Marisa Wexler, MS
Beyond Batten Disease Foundation (BBDF) has struck a deal with Actelion Pharmaceuticals to help further the development of BBDF-101,…
New guidelines from the U.S. Food and Drug Administration (FDA) may make it easier to develop therapies to treat…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
A patient with both Batten disease and Moyamoya disease — a condition where blood vessels in the skull…
Abeona Therapeutics and Taysha Gene Therapies have entered into agreements concerning ABO-202, an investigative gene therapy for…
The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation and rare pediatric disease designation…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a gene therapy being developed by …
The number of fluid-containing cellular compartments called vacuoles within immune cells could help to predict the severity of…